Gene Therapy Enables Children With Rare Disorder to Walk, Talk for First Time

Children with AADC deficiency experience severe motor dysfunction, inability to walk or speak, and involuntary eye spasms; gene therapy has restored these capabilities in some patients.

In a surgical trial in Germany, thirty children born with a disorder so rare it has touched fewer than 120 lives worldwide received a gene therapy delivered directly into the brain — and every one of them improved. The therapy, Upstaza, restored what a single broken gene had withheld: the neurochemical machinery of movement and speech. Seven children walked for the first time; three spoke their first words. In approving it as the first brain-infused gene therapy in Europe, medicine has quietly crossed a threshold that once belonged only to imagination.

Gene Therapy Enables Children With Rare Disorder to Walk, Talk for First Time

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